New Data Show ELOCTATE® and ALPROLIX® May Help Control Target Joint Bleeds in People with Hemophilia A and B

New data demonstrate ELOCTATE^® [Antihemophilic Factor (Recombinant), Fc Fusion Protein] (marketed as ELOCTA^® in Europe) and ALPROLIX^® [Coagulation Factor IX (Recombinant), Fc Fusion Protein] may effectively manage target joint bleeding and maintain low annualized bleeding rates (ABRs) in people with severe hemophilia A and B. The data, which were presented by Biogen (NASDAQ:BIIB) and Swedish Orphan Biovitrum AB (publ) (Sobi) (STO: SOBI) at the 57^th American Society of Hematology (ASH) Annual Meeting and Exposition in Orlando, Fla., continue to reinforce the value of extended interval prophylactic dosing of ELOCTATE and ALPROLIX.

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“As the first prolonged half-life therapies, ELOCTATE and ALPROLIX have shown low rates in both joint bleeding and overall annualized bleeding episodes,” said Kate Dawson M.D., vice president, U.S. Medical at Biogen. “Their ability to reduce bleed rates, which may translate into the potential for reducing some joint disease, continues to reaffirm their clinical value for people living with hemophilia A and B.”

For people with severe hemophilia A and B, most bleeding events occur in joints. When bleeding events occur repeatedly in the same joint (known as a target joint), it is often a precursor to chronic joint disease, marked by progressive deterioration of the joint.¹ These post-hoc analyses aimed to assess the frequency of bleeding events and the dosing of ELOCTATE and ALPROLIX in study participants who had one or more target joint bleeds [major joint (e.g., knee, elbow, ankle) with three or more bleeding episodes in a three-month (hemophilia B) or six-month (hemophilia A) period].^2,3

“Understanding the impact of ELOCTATE and ALPROLIX on people with target joint bleeds provides further insight into their value in a real-world setting,” said Birgitte Volck, M.D., Ph.D., senior vice president of Development and chief medical officer of Sobi. “These new results from the post hoc analyses highlight the value of extended half-life therapy in managing and controlling bleeds, adding to the body of robust clinical data and the longest real-world experience of any extended half-life therapy to date."

Results Highlight Therapies’ Potential for Reducing Bleed Rates in Target Joints
In this ASPIRE (an ongoing extension of Phase 3 pivotal trials A-LONG and Kids A-LONG) post-hoc analysis, for people with hemophilia A taking ELOCTATE prophylactically, on-study annualized bleeding rates (ABRs) overall and in target joints were lower than pre-study bleeding rates. Data from ASPIRE showed that nearly half of the adult and adolescent participants in the weekly prophylaxis, individualized prophylaxis and modified prophylaxis arms (n=26, n=82, n=12, respectively) did not have any target joint bleeds (42.3, 47.6 and 41.7 percent, respectively). For children, 53.8 percent of participants in the individualized prophylaxis group (n=13) did not have any target joint bleeding episodes. In addition, nearly all (97.4 percent) target joints in adult and adolescent participants taking ELOCTATE were resolved during the follow-up period, suggesting that the therapy may be equally effective for preventing target joint bleeding episodes in weight-bearing and non-weight-bearing joints. The median dosing intervals for ASPIRE participants with target joints at baseline were similar to those for the A-LONG and Kids A-LONG overall population.²

In the B-LONG (the pivotal Phase 3 study) post-hoc analysis, for people with hemophilia B taking ALPROLIX prophylactically, the therapy was shown effective in reducing the frequency of bleeding episodes overall and in target joints. The analysis found that 48.6 percent of participants receiving weekly prophylaxis (n=35) and 37.5 percent of participants on individualized interval prophylaxis (n=8) did not have any target joint bleeds at the end of B-LONG. Overall, participants’ target joint, spontaneous target joint and traumatic target joint median ABRs were low for participants in the weekly prophylaxis arm (1.03, 0.00 and 0.00 respectively) and the individualized interval prophylaxis arm (2.20, 2.20 and 0.00 respectively). Additionally, among B-LONG participants entering the trial with target joints (n=43), the on-study median dosing intervals were longer (6.98 days in the weekly prophylaxis arm and 10.25 in the individualized interval prophylaxis arm) than the pre-study dosing interval with a traditional factor therapy, suggesting that target joint bleeds may be effectively managed and controlled with an extended prophylactic dosing regimen.³

About Hemophilia A and B
Hemophilia is a rare, genetic disorder in which the ability of a person’s blood to clot is impaired. Hemophilia A occurs in about one in 5,000 male births annually, and more rarely in females. Hemophilia B occurs in about one in 25,000 male births annually, and more rarely in females. Worldwide, it is estimated that more than 400,000 people are living with hemophilia. Hemophilia A is caused by having substantially reduced or no factor VIII activity, while hemophilia B is caused by having substantially reduced or no factor IX activity; factor VIII and factor IX are needed for normal blood clotting.

People with hemophilia A or B experience prolonged bleeding episodes that can cause pain, irreversible joint damage and life-threatening hemorrhages. Prophylactic infusions of factor VIII or IX can temporarily replace the missing clotting factors that are needed to control bleeding and prevent new bleeding episodes.⁴ The Medical and Scientific Advisory Council of the National Hemophilia Foundation recommends prophylaxis as the optimal therapy for people with severe hemophilia A or B.⁵

About ELOCTATE/ELOCTA
ELOCTATE^® [Antihemophilic Factor (Recombinant), Fc Fusion Protein], the first recombinant clotting factor VIII therapy with prolonged circulation in the body, is approved in the United States, Canada, Australia and Japan. In the European Union, it was recently approved and marketed under the trade name ELOCTA^®. It is indicated in the United States for the control and prevention of bleeding episodes, perioperative (surgical) management and routine prophylaxis in adults and children with hemophilia A. ELOCTATE is not indicated for the treatment of a bleeding disorder called von Willebrand disease. ELOCTATE was developed by fusing B-domain deleted factor VIII to the Fc portion of immunoglobulin G subclass 1, or IgG₁ (a protein commonly found in the body). It is believed that this enables ELOCTATE to use a naturally occurring pathway to prolong the time the therapy remains in the body.

Common adverse reactions (incidence of greater than or equal to 1 percent) reported in the registrational A-LONG study were arthralgia (joint pain) and malaise (general discomfort). For important safety information, and the United States full prescribing information, please visit www.ELOCTATE.com.

About ALPROLIX
ALPROLIX^® [Coagulation Factor IX (Recombinant), Fc Fusion Protein], the first recombinant clotting factor therapy with prolonged circulation in the body, is approved in the United States, Canada, Australia and Japan. It is indicated in the United States for the control and prevention of bleeding episodes, perioperative (surgical) management and routine prophylaxis in adults and children with hemophilia B. ALPROLIX is not indicated for immune tolerance induction therapy, which is a treatment for people with inhibitors, and should not be used in individuals with a known history of serious allergic reactions. ALPROLIX was developed by fusing factor IX to the Fc portion of immunoglobulin G subclass 1, or IgG₁. It is believed that this enables ALPROLIX to use a naturally occurring pathway to prolong the time the therapy remains in the body.

Common adverse reactions (incidence of greater than or equal to 1 percent) from the registrational B-LONG study were headache and oral paresthesia (an abnormal sensation in the mouth). For additional important safety information, and the United States full prescribing information, please visit www.ALPROLIX.com.

About Biogen
Through cutting-edge science and medicine, Biogen discovers, develops and delivers worldwide innovative therapies for people living with serious neurological, autoimmune and rare diseases. Founded in 1978, Biogen is one of the world’s oldest independent biotechnology companies and patients worldwide benefit from its leading multiple sclerosis and innovative hemophilia therapies. For more information, please visit www.biogen.com. Follow us on Twitter.

About Sobi
Sobi is an international specialty healthcare company dedicated to rare diseases. Sobi’s mission is to develop and deliver innovative therapies and services to improve the lives of patients. The product portfolio is primarily focused on Haemophilia, Inflammation and Genetic diseases. Sobi also markets a portfolio of specialty and rare disease products for partner companies across Europe, the Middle East, North Africa and Russia. Sobi is a pioneer in biotechnology with world-class capabilities in protein biochemistry and biologics manufacturing. In 2014, Sobi had total revenues of SEK 2.6 billion (USD 380 M) and about 600 employees. The share (STO: SOBI) is listed on NASDAQ OMX Stockholm. More information is available at www.sobi.com.

Biogen Safe Harbor
This press release contains forward-looking statements, including statements about the potential benefits and efficacy of ELOCTATE and ALPROLIX. These statements may be identified by words such as “believe,” “expect,” “may,” “plan,” “potential,” “will” and similar expressions, and are based on our current beliefs and expectations. Drug development and commercialization involve a high degree of risk. Factors which could cause actual results to differ materially from our current expectations include the risk that unexpected concerns may arise from additional data or analysis, regulatory authorities may require additional data or information or further studies, or may fail to approve, or refuse to approve, or may delay approval of our drug candidates, or we may encounter other unexpected hurdles. For more detailed information on the risks and uncertainties associated with our drug development and commercialization activities, please review the Risk Factors section of our most recent annual or quarterly report filed with the Securities and Exchange Commission. Any forward-looking statements speak only as of the date of this press release and we assume no obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise.

References:

1. Hemophilia Federation of America. Joint Damage. Available at: http://www.hemophiliafed.org/bleeding-disorders/complications/joint-damage/. Accessed: November 2015.

2. Kerlin, BA, et al. (December 2015). Long-term Efficacy of rFVIIIFc Prophylaxis in Pediatric, Adolescent, and Adult Subjects With Target Joints and Severe Hemophilia A. Poster session presented at the American Society of Hematology (ASH) Annual Meeting and Exposition in Orlando, Fla.

3. Shapiro, AD, et al. (December 2015). Analysis of Target Joint Bleeding With Prophylactic Use of Recombinant Factor IX Fc Fusion Protein in Patients With Severe Hemophilia B. Poster session presented at the American Society of Hematology (ASH) Annual Meeting and Exposition in Orlando, Fla.

4. Hemophilia Federation of America. What is Hemophilia? Available at: http://www.hemophiliafed.org/bleeding-disorders/hemophilia/treatment/ . Accessed: November 15.

5. Hemophilia Federation of America. MASAC Recommendation Concerning Prophylaxis. Available at: https://www.hemophilia.org/Researchers-Healthcare-Providers/Medical-and-Scientific-Advisory-Council-MASAC/MASAC-Recommendations/MASAC-Recommendation-Concerning-Prophylaxis . Accessed: November 2015.

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